On November 3, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and third quarter 2022 financial results (Press release, Sangamo Therapeutics, NOV 3, 2022, View Source [SID1234622961]).
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"This has been a year marked by progress across our pipeline. In the third quarter, we continued to advance our clinical trials and preclinical activities while maintaining fiscal discipline and operational excellence," said Sandy Macrae, Chief Executive Officer of Sangamo. "We were proud to present promising updated preliminary data from our wholly owned Fabry study, to resume our partnered Hemophilia A pivotal trial, and to continue dosing in our renal transplant rejection and sickle cell studies. Our pipeline progress is expected to yield additional data in Q4 and into 2023. As we look to next year and beyond, I am confident in Sangamo’s ability to carry out our mission of developing transformational therapies for patients in need."
Recent Business Highlights
Fabry disease – Reported data updates from the Phase 1/2 STAAR study’s dose escalation phase; Dose expansion phase underway and dosing commenced; Phase 3 planning progresses.
Presented updated preliminary data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry Disease at three separate conferences, most notably at the 29th Congress of the European Society of Gene & Cell Therapy (ESGCT), presenting updated data as of July 21, 2022.
Preliminary data showed all nine patients from the dose escalation phase exhibited sustained elevated α-Gal A activity, ranging from nearly 2-fold to 30-fold of mean normal, for up to 23 months post dosing, as of the last date of measurement.
Four patients were withdrawn from enzyme replacement therapy (ERT) and maintained significantly elevated levels of α-Gal A activity up to 28 weeks post withdrawal. Since the cutoff date, the fifth and final patient in the dose escalation phase who started the study on ERT has been withdrawn from ERT. All patients withdrawn have remained off ERT.
The Phase 1/2 STAAR study has transitioned into the expansion phase, with the first five expansion patients dosed at the 5e13 vg/kg dose level, including the first two female patients.
We expect to present additional clinical updates from the STAAR study, including the first data from the expansion cohort, in the first half of 2023.
We continue to actively prepare for a potential pivotal Phase 3 trial.
Sickle cell disease – Dosed sixth patient, the second with a product candidate manufactured using improved methods; Phase 3 planning progresses.
We dosed the sixth patient in the Phase 1/2 PRECIZN-1 study of BIVV003, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease. This is the second patient in the study to receive a product candidate manufactured using improved methods that have been shown in internal experiments to increase the number of long-term progenitor cells in the final product.
Received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BIVV003.
We expect to present updated data from the Phase 1/2 PRECIZN-1 study via a poster presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition on December 10-13, 2022 in New Orleans, Louisiana.
Phase 3 study design, enabling activities and manufacturing readiness are in progress.
Renal Transplant Rejection – Dosed the second patient in the Phase 1/2 STEADFAST study; progressed clinical activities in preparation for patient three.
Dosed the second patient in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
The product candidate continues to be generally well tolerated in both patients.
Progressed clinical activities in preparation for the third patient.
We plan to provide guidance on timing for dosing for the third patient once the kidney transplant has been scheduled.
Hemophilia A – Announced, with Pfizer, the resumption of recruitment in the Phase 3 AFFINE trial; dosing is expected to resume shortly; pivotal data read-out expected in the first half of 2024.
Pfizer and Sangamo announced that recruitment has re-opened in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A.
Trial sites resumed enrollment in September, and dosing is expected to resume shortly.
A pivotal readout is expected in the first half of 2024.
We expect to present updated data from the Phase 1/2 ALTA study via a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December.
Third Quarter 2022 Financial Results
Consolidated net loss for the third quarter ended September 30, 2022 was $53.2 million, or $0.34 per share, compared to a net loss of $47.7 million, or $0.33 per share, for the same period in 2021.
Revenues
Revenues for the third quarter ended September 30, 2022 were $26.5 million, compared to $28.6 million for the same period in 2021.
The decrease of $2.1 million in revenues was primarily attributable to a decrease of $1.9 million and $1.6 million related to our collaboration agreements with Novartis and Biogen respectively, and a decrease of $1.1 million due to the termination of our collaboration agreement with Sanofi. These decreases were partially offset by a $1.9 million adjustment to revenue during 2021 related to the collaboration agreement with Sanofi and an increase of $0.5 million in revenue related to our collaboration agreement with Kite.
Total operating expenses on a GAAP basis for the third quarter ended September 30, 2022 were $81.3 million, compared to $77.0 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the third quarter ended September 30, 2022 were $73.5 million, compared to $69.1 million for the same period in 2021.
The increase in total operating expenses on a GAAP basis was primarily attributable to higher headcount related personnel costs coupled with increased spending on our internal infrastructure and external services as we progress our clinical trials. These increases were partially offset by reimbursement of certain research and development expenses by Sanofi under the termination agreement.
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities as of September 30, 2022, were $350.3 million, compared to $464.7 million as of December 31, 2021. We have raised approximately $75.1 million in net proceeds under our at-the-market offering program from January 1, 2022 through October 31, 2022.
Financial Guidance for 2022 Narrowed (initial guidance provided on February 24, 2022)
On a GAAP basis, we expect our total operating expenses which includes non-cash stock-based compensation expenses, to be lower than previously guided and be in the range of approximately $315 million to $325 million.
We expect our non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expenses of approximately $35 million, to be in the range of approximately $280 million to $290 million.
Upcoming Events
Sangamo plans to participate in the following events in the fourth quarter:
Scientific / Medical Conferences
64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, December 10-13, New Orleans, Louisiana
Investor Conferences
31st Annual Credit Suisse Healthcare Conference, November 8, 2022
Barclays Gene Editing & Gene Therapy Summit, November 14, 2022
Stifel Healthcare Conference, November 15, 2022
Jefferies London Healthcare Conference, November 16, 2022
EvercoreISI HealthCONxConference, November 29 – December 1, 2022
Access links for available webcasts for these investor conferences will be available on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Available materials will be found on the Sangamo Therapeutics website after the event.
Conference Call to Discuss Third Quarter 2022 Results
The Sangamo management team will discuss these results on a conference call today, Thursday November 3, 2022, at 4:30 p.m. Eastern Time.
Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.
The link to access the live webcast can also be found on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.
A replay will be available following the conference call, accessible under Events and Presentations.